CBER 2026 Orphan Approvals (new BLAs)
This list reflects information regarding the applications as of the approval date. It is not updated with regard to applicant or application status changes. The applications are listed by date of approval in reverse chronological order.
| Trade Name/Proper Name | Indication | Manufacturer/License Number | Approval Date |
|---|---|---|---|
| KRESLADI | for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. | Rocket Pharmaceuticals,RInc | 3/26/2026 |